Biohaven’s drug for genetic neurological disease fails late-stage study

Biohaven Pharma said recently its experimental drug for patients with spinocerebellar ataxia, a genetic disease that affects the nervous system, failed to meet the main goal of a late-stage study.

Spinocerebellar ataxia (SCA) is a progressive disorder caused by the degeneration of cells in the brain and spinal cord and can lead to symptoms such as uncoordinated movement and muscle wasting. It has no approved treatments in the US.

The main goal in the Biohaven study measured the change in severity of symptoms among patients who took the drug, troriluzole, from the baseline to week 48 of treatment.

Biohaven said patients in both the drug and placebo group showed a less-than-expected change in disease progression, which led to the study not reaching statistical significance.

But troriluzole showed some benefit compared with placebo in patients with SCA Type-III – the most common form of the disease, Biohaven said.

The company plans to share the data on the subtype with regulators and said it would work with the US Food and Drug Administration (FDA) on a path forward for the drug.

Biohaven earlier this month agreed to be bought by Pfizer in a $11.6 billion deal that underscored the buyer’s confidence in its commercial migraine drug.

Separately, the company said the FDA has accepted the marketing application for its zavegepant nasal spray to treat migraine in adults, with a decision expected in the first quarter of 2023.

Edits by EP News Bureau